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EP3562494A4 - GENE THERAPY FOR TREATMENT OF PHENYLKETONURIA - Google Patents

GENE THERAPY FOR TREATMENT OF PHENYLKETONURIA Download PDF

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Publication number
EP3562494A4
EP3562494A4 EP17887983.9A EP17887983A EP3562494A4 EP 3562494 A4 EP3562494 A4 EP 3562494A4 EP 17887983 A EP17887983 A EP 17887983A EP 3562494 A4 EP3562494 A4 EP 3562494A4
Authority
EP
European Patent Office
Prior art keywords
phenylketonuria
treatment
gene therapy
therapy
gene
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP17887983.9A
Other languages
German (de)
French (fr)
Other versions
EP3562494A1 (en
Inventor
James M. Wilson
Jenny Agnes SIDRANE
Scott ASHLEY
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Pennsylvania Penn
Original Assignee
University of Pennsylvania Penn
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of Pennsylvania Penn filed Critical University of Pennsylvania Penn
Publication of EP3562494A1 publication Critical patent/EP3562494A1/en
Publication of EP3562494A4 publication Critical patent/EP3562494A4/en
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Obesity (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Environmental Sciences (AREA)
  • Mycology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
EP17887983.9A 2016-12-30 2017-12-29 GENE THERAPY FOR TREATMENT OF PHENYLKETONURIA Pending EP3562494A4 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662440651P 2016-12-30 2016-12-30
US201762469898P 2017-03-10 2017-03-10
US201762505373P 2017-05-12 2017-05-12
PCT/US2017/068897 WO2018126112A1 (en) 2016-12-30 2017-12-29 Gene therapy for treating phenylketonuria

Publications (2)

Publication Number Publication Date
EP3562494A1 EP3562494A1 (en) 2019-11-06
EP3562494A4 true EP3562494A4 (en) 2020-08-19

Family

ID=62709967

Family Applications (1)

Application Number Title Priority Date Filing Date
EP17887983.9A Pending EP3562494A4 (en) 2016-12-30 2017-12-29 GENE THERAPY FOR TREATMENT OF PHENYLKETONURIA

Country Status (8)

Country Link
US (2) US11382941B2 (en)
EP (1) EP3562494A4 (en)
JP (1) JP7229161B2 (en)
KR (1) KR102738752B1 (en)
CN (1) CN110325199A (en)
BR (1) BR112019013576A2 (en)
MX (1) MX2019007876A (en)
WO (1) WO2018126112A1 (en)

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WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
US11980663B2 (en) 2015-07-08 2024-05-14 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
IL279520B2 (en) 2016-01-15 2023-03-01 American Gene Tech Int Inc Methods and compositons for the activation of gamma-delta t-cells
WO2017139065A1 (en) 2016-02-08 2017-08-17 American Gene Technologies International Inc. Hiv vaccination and immunotherapy
EP3426777B1 (en) 2016-03-09 2022-02-16 American Gene Technologies International Inc. Combination vectors and methods for treating cancer
WO2017213697A1 (en) 2016-06-08 2017-12-14 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
EP3481418A4 (en) 2016-07-08 2020-03-11 American Gene Technologies International Inc. HIV PRE-IMMUNIZATION AND IMMUNOTHERAPY
EP3487507A4 (en) 2016-07-21 2020-04-08 American Gene Technologies International, Inc. VIRAL VECTORS FOR TREATING PARKINSON'S DISEASE
MX2019000962A (en) 2016-07-26 2019-08-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins.
EP3607072A4 (en) 2017-04-03 2021-01-06 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
MA50801A (en) 2017-11-22 2020-09-30 Modernatx Inc POLYNUCLEOTIDES CODING FOR PHENYLALANINE HYDROXYLASE FOR THE TREATMENT OF PHENYLKETONURIS
PE20212076A1 (en) 2018-02-01 2021-10-26 Homology Medicines Inc ADENO-ASSOCIATED VIRUS COMPOSITIONS TO RESTORE THE FUNCTION OF THE PAH GENE AND METHODS OF USE OF THE SAME
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
BR112020015511A2 (en) * 2018-02-01 2021-01-26 Homology Medicines, Inc. adeno-associated virus compositions for gene transfer from pah and methods of using them
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
TW202005978A (en) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 Novel liver targeting adeno-associated viral vectors
US12472268B2 (en) 2018-10-01 2025-11-18 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
US20210348135A1 (en) * 2018-10-12 2021-11-11 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
EP3938515A4 (en) * 2019-03-13 2022-12-28 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) therapeutics
CA3137698A1 (en) * 2019-05-31 2020-12-03 Tyler LAHUSEN Optimized phenylalanine hydroxylase expression
AU2020372988A1 (en) * 2019-10-30 2023-06-01 Amicus Therapeutics, Inc. Recombinant CDKL5 proteins, gene therapy and production methods
TW202140791A (en) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 Methods of treating phenylketonuria
EP4090382A2 (en) * 2020-01-16 2022-11-23 Takeda Pharmaceutical Company Limited Adeno associated virus based gene therapy for phenylketonuria
WO2021202532A1 (en) * 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
AR122409A1 (en) 2020-04-03 2022-09-07 Biomarin Pharm Inc TREATMENT OF PHENYLKETONURIA WITH AAV AND THERAPEUTIC FORMULATIONS
TW202208632A (en) 2020-05-27 2022-03-01 美商同源醫藥公司 Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
WO2021252952A1 (en) * 2020-06-11 2021-12-16 Sangamo Therapeutics, Inc. Methods and compositions for expressing phenylalanine hydroxylase
KR20230066615A (en) * 2020-09-16 2023-05-16 제너레이션 바이오 컴퍼니 Closed DNA vectors and their use for phenylalanine hydroxylase (PAH) expression
JP2023544165A (en) * 2020-10-01 2023-10-20 ジェンザイム・コーポレーション Human PAH expression cassette for treating PKU by liver-directed gene replacement therapy
CA3209779A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
CN113265424B (en) * 2021-05-14 2022-07-05 华中科技大学同济医学院附属协和医院 Recombinant adeno-associated virus vector for treating atherosclerosis, genome composition and application
WO2025217184A1 (en) * 2024-04-08 2025-10-16 The Trustees Of The University Of Pennsylvania Compositions and methods for in vivo nuclease-mediated treatment of phenylketonuria (pku)
CN119876192B (en) * 2024-07-02 2025-11-07 浙江大学滨江研究院 Polynucleotide for treating phenylketonuria

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See also references of WO2018126112A1 *

Also Published As

Publication number Publication date
US11382941B2 (en) 2022-07-12
US20190336550A1 (en) 2019-11-07
CA3048038A1 (en) 2018-07-05
EP3562494A1 (en) 2019-11-06
WO2018126112A1 (en) 2018-07-05
JP7229161B2 (en) 2023-02-27
JP2020504619A (en) 2020-02-13
KR102738752B1 (en) 2024-12-06
KR20190100318A (en) 2019-08-28
BR112019013576A2 (en) 2020-02-04
US20220313760A1 (en) 2022-10-06
MX2019007876A (en) 2019-10-15
CN110325199A (en) 2019-10-11

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